Table 1.
Characteristic of patients, disease, and treatment
| Variable | Total treated cohort, N = 19 |
Allogeneic SCT cohort, n = 11 |
Maintenance therapy cohort, n = 8 |
P | |||
|---|---|---|---|---|---|---|---|
| N or median | % or IQR | n or median | % or IQR | n or median | % or IQR | ||
| Male | 6 | 31.60% | 3 | 27.30% | 3 | 37.5% | 1 |
| Age at CAR T-cell infusion, y | 7.9 | 6.1-12.6 | 7.9 | 6.3-12.8 | 8.75 | 4.9-12.2 | .8043 |
| Characteristics of initial diagnosis | |||||||
| Infant ALL, n | 3 | 15.80% | 1 | 9.10% | 2 | 25% | .5459 |
| Median WCC (x 109/L) | 10 | 9.4-99.8 | 10 | 9.9-44 | 40 | 8.5-128.5 | .8729 |
| NCI risk | |||||||
| High | 7 | 36.80% | 2 | 27.30% | 4 | 50% | .37 |
| Standard | 9 | 47.40% | 7 | 54.50% | 3 | 37.50% | |
| Not known | 3 | 15.80% | 2 | 18.20% | 1 | 12.50% | |
| Cytogenetic risk | |||||||
| Good risk (total) | 9 | 47.40% | 5 | 45.50% | 4 | 50% | 1 |
| G ood risk (ETV6-RUNX1) | 3 | 15.80% | 2 | 18.20% | 1 | 12.50% | |
| Good risk (high hyperdiploid) | 6 | 31.60% | 3 | 27.30% | 3 | 37.50% | |
| Intermediate risk (total) | 1 | 5.30% | 1 | 9.10% | 0 | ||
| Intermediate risk (IKZF1 deletion) | 1 | 5.30% | 1 | 9.10% | 0 | ||
| High risk (total) | 5 | 26.30% | 3 | 27.30% | 2 | 25% | |
| High risk (KMT2Ar) | 3 | 15.80% | 1 | 9.10% | 2 | 25% | |
| High risk (BCR-ABL) | 1 | 5.30% | 1 | 9.10% | 0 | ||
| High risk (t(17;19)/TCF3-HLF) | 1 | 5.30% | 1 | 9.10% | 0 | ||
| Uninformative cytogenetics | 4 | 21.10% | 2 | 18.20% | 2 | 25% | |
| Unknown | 1 | 5.30% | 1 | 9.10% | 0 | ||
| Other | 3 | 15.80% | 1 | 9.10% | 2 | 25% | |
| Characteristics of relapsed/refractory disease | |||||||
| Indication for CAR T-cell therapy | |||||||
| Primary refractory | 1 | 5.30% | 1 | 9.10% | 0 | 1 | |
| Relapse | 18 | 94.70% | 10 | 90.90% | 8 | 100% | |
| Refractory status at any time point | 11 | 57.90% | 7 | 63.60% | 4 | 50% | .6577 |
| Prior therapy | |||||||
| Median number of relapses | 1 | 1.0-2.0 | 1 | 1.0-1.5 | 1.5 | 1.0-2.0 | .2754 |
| CNS and extramedullary disease | |||||||
| CNS at any point | 11 | 57.90% | 5 | 45.50% | 6 | 75% | .3521 |
| EM non-CNS relapses (both isolated and combined) | 2 | 10.50% | 1 | 9.10% | 1 | 12.50% | 1 |
| Median number of previous therapy lines (excluding SCT) | 2 | 2.0-2.0 | 2 | 2.0-2.0 | 2 | 2.0-2.2 | .7737 |
| Prior SCT | 5 | 26.30% | 2 | 18.20% | 3 | 37.50% | .6027 |
| Blinatumomab exposure | 4 | 21.10% | 1 | 9.10% | 3 | 37.50% | .2621 |
| Inotuzumab exposure | 1 | 5.30% | 0 | 1 | 12.50% | .4211 | |
| CAR T-cell therapy | |||||||
| Status before lymphodepletion | |||||||
| High disease burden (≥5%) | 3 | 15.80% | 2 | 18.20% | 1 | 12.50% | 1 |
| Low disease burden (<5%) | 11 | 57.90% | 6 | 54.60% | 5 | 62.50% | |
| Undetectable by MRD | 5 | 26.30% | 3 | 27.30% | 2 | 25% | |
| CAR T-cell product | |||||||
| Tisagenlecleucel | 17 | 89.50% | 10 | 90.90% | 7 | 87.50% | 1 |
| Experimental | 2 | 10.50% | 1 | 9.10% | 1 | 12.50% | |
| Median CAR T-cell dose, cells per kg, (n = 17) | 2.7 × 106 | 1.8-3.7 x 106 | 3.4 × 106 | 2.5-4.3 x 106 | 2.4 × 106 | 1.5-2.8 x 106 | .2069 |
| Time to LBCA | |||||||
| Median (months) | 2.66 | 95% CI : 2.3-3.6 | 2.66 | 95% CI : 2.07 to NR | 2.64 | 95% CI : 2.3 to NR | .9 |
| 0-3 months | 13 | 68.4% | 7 | 63.60% | 6 | 75% | 1 |
| 3-6 months | 6 | 31.60% | 4 | 34.4% | 2 | 25% | |
| Post–CAR T-cell treatment | |||||||
| Median time from LBCA to SCT (mo) | 2.4 | 1.7-4.5 | 1 | ||||
| SCT conditioning | |||||||
| TBI-based | 8 | 72.7% | |||||
| Non-TBI | 3 | 27.30% | |||||
| SCT donor | |||||||
| MUD | 7 | 72.70% | |||||
| MMUD (UCB) | 1 | 9.00% | |||||
| MSD | 2 | 18.20% | |||||
| Haplo | 1 | 9.00% | |||||
| Maintenance with VCR/Dexa pulses | |||||||
| Yes | 4 | 50% | |||||
| No | 4 | 50% | |||||
CNS, central nervous system; Dexa, dexamethasone; EM, extramedullary; Haplo, haploidentical; IQR, interquartile range; MMUD, mismatched unrelated donor; MRD minimal residual disease; MSD, matched sibling donor; MUD, matched unrelated donor; NCI, National Cancer Institute; NR, not reached; TBI, total body irradiation; UCB umbilical cord blood; VCR, vincristine; WCC, white blood cell count.